Effect of dose and plasma concentration on liver uptake and pharmacologic activity of a 2′-methoxyethyl modified chimeric antisense oligonucleotide targeting PTEN

Biochemical Pharmacology

Volumn 78, Issue 3, 2009, Pages 284-291

  Geary, Richard S ^a   Wancewicz, Ed ^a   Matson, John ^a   Pearce, Megan ^a   Siwkowski, Andrew ^a   Swayze, Eric ^a   Bennett, Frank ^a  

^a ISIS PHARMACEUTICALS   (United States)

Author keywords

Antisense oligonucleotide; Liver; mRNA; Pharmacodynamics; Pharmacokinetics

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ISIS 116847; ISIS 13920; MESSENGER RNA; PHOSPHATIDYLINOSITOL 3,4,5 TRISPHOSPHATE 3 PHOSPHATASE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BOLUS INJECTION; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG ACTIVITY; DRUG BLOOD LEVEL; DRUG ELIMINATION; DRUG INFUSION; DRUG INHIBITION; DRUG LIVER LEVEL; DRUG METABOLISM; DRUG UPTAKE; LIVER METABOLISM; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL;

ANIMALS; BASE SEQUENCE; DNA PRIMERS; HALF-LIFE; LIVER; MALE; MICE; MICE, INBRED BALB C; OLIGONUCLEOTIDES, ANTISENSE; PTEN PHOSPHOHYDROLASE; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION;

EID: 65749101352     PISSN: 00062952     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.bcp.2009.04.013     Document Type: Article

References (48)

1. Wagner R.W. Gene inhibition using antisense oligodeoxynucleotides. Nature 372 (1994) 333-335
2. Altmann K.-H., Dean N.M., Fabbro D., Freier S.M., Geiger T., Haner R., et al. Second generation of antisense oligonucleotides: from nuclease resistance to biological efficacy in animals. Chimia 50 (1996) 168-176
3. Lima W.F., Venkatraman M., and Crooke S.T. The influence of antisense oligonucleotide-induced RNA structure on E. coli RNase H1 activity. J Biol Chem 272 (1997) 18191-18199
4. Manoharan M. 2′-Carbohydrate modifications in antisense oligonucleotide therapy: importance of conformation, configuration and conjugation. Biochim Biophys Acta 1489 (1999) 117-130
5. McKay R.A., Miraglia L.J., Cummins L.L., Owens S.R., Sasmor H., and Dean N.M. Characterization of a potent and specific class of antisense oligonucleotide inhibitor of human protein kinase C-α expression. J Biol Chem 274 (1999) 1715-1722
6. Henry S.P., Geary R.S., Yu R., and Levin A.A. Drug properties of second-generation antisense oligonucleotides: how do they measure up to their predecessors?. Curr Opin Invest Drugs 2 (2001) 1444-1449
7. Sewell L.K., Geary R.S., Baker B.F., Glover J.M., Mant T.G.K., Yu R.Z., et al. Phase I trial of ISIS 104838, a 2′-methoxyethyl modified antisense oligonucleotide targeting tumor necrosis factor-alpha. J Pharmacol Exp Ther 303 (2002) 1334-1343
8. Wei N., Fiechtner J., Boyle D., Kavanaugh A., Delauter S., Rosengren S., et al. Synovial Biomarker Study of ISIS 104838, an antisense oligodeoxynucleotide targeting TNF-alpha, in rheumatoid arthritis. 67th annual meeting of the American College of Rheumatology (ACR). Orlando, FL (2003)
9. Kjems L. New targets for glycemic control protein-tyrosine-phosphatase-1B antisense inhibitor. The American Diabetes Association's 65th annual meeting. San Diego, CA (2005)
10. Chi K.N., Eisenhauer E., Fazli L., Jones E.C., Goldenberg S.L., Powers J., et al. A phase I pharmacokinetic and pharmacodynamic study of OGX-011, a 2′methoxyethyl antisense to clusterin, in patients with localized prostate cancer prior to radical prostatectomy. The 40th annual meeting of the American Society of Clinical Oncology. New Orleans, LA (2004)
11. Jones B.A., and Schreiber A.D. mRNA as a therapeutic target in lung disease. Drug Des Rev 2 (2005) 361-372
12. ATL 1102: a new therapeutic option for RRMS? Inpharma 2008;1646:12.
13. Kastelein J.J., Wedel M.K., Baker B.F., Su J., Bradley J.D., Yu R.Z., et al. Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation 114 (2006) 1729-1735
14. Geary R.S., Ushiro-Watanabe T., Truong L., Freier S.M., Lesnik E.A., Sioufi N.B., et al. Pharmacokinetic properties of 2′-O-(2-methoxyethyl)-modified oligonucleotide analogs in rats. J Pharmacol Exp Ther 296 (2001) 890-897
15. Geary R.S., Yu R.Z., Watanabe T., Henry S.P., Hardee G.E., Chappell A., et al. Pharmacokinetics of a tumor necrosis factor-alpha phosphorothioate 2′-O-(2-methoxyethyl) modified antisense oligonucleotide: comparison across species. Drug Metab Dispos 31 (2003) 1419-1428
16. Yu R.Z., Kim T.-W., Hong A., Watanabe T.A., Gaus H.J., and Geary R.S. Cross-species pharmacokinetic comparison from mouse to man of a second generation antisense oligonucleotide ISIS 301012, targeting human ApoB-100. Drug Metab Dispos 35 (2007) 460-468
17. Levin A.A., Yu R.Z., and Geary R.S. Basic principles of the pharmacokinetics of antisense oligonucleotide drugs. In: Crooke S.T. (Ed). Antisense drug technology, principles, strategies and applications (2007), Taylor & Francis Group, Boca Raton, FL 183-215
18. Geary R.S., Leeds J.M., Henry S.P., Monteith D.K., and Levin A.A. Antisense oligonucleotide inhibitors for the treatment of cancer. 1. Pharmacokinetic properties of phosphorothioate oligodeoxynucleotides. Anticancer Drug Des 12 (1997) 383-393
19. Crooke R.M. Cellular uptake, distribution and metabolism of phosphorothioate, phosphodiester and methylphosphonate oligonucleotides. In: Crooke S.T., and Lebleu B. (Eds). Antisense research and applications (1993), CRC Press, Boca Raton, FL 427-449
20. Beltinger C., Saragovi H.U., Smith R.M., LeSauteur L., Shah N., DeDionisio L., et al. Binding, uptake, and intracellular trafficking of phosphorothioate-modified oligodeoxynucleotides. J Clin Invest 95 (1995) 1814-1823
21. Crooke R.M., Graham M.J., Cooke M.E., and Crooke S.T. In vitro pharmacokinetics of phosphorothioate antisense oligonucleotides. J Pharmacol Exp Ther 275 (1995) 462-473
22. Bijsterbosch M.K., Manoharan M., Rump E.T., De Vrueh R.L.A., van Veghel R., Tival K.L., et al. In vivo fate of phosphorothioate antisense oligodeoxynucleotides: predominant uptake by scavenger receptors on endothelial cells. Nucleic Acids Res 25 (1997) 3290-3296
23. Butler M., Crooke R.M., Graham M.J., Lemonidis K.M., Lougheed M., Murray S.F., et al. Phosphorothioate oligodeoxynucleotides distribute similarly in class A scavenger receptor knockout and wild-type mice. J Pharmacol Exp Ther 292 (2000) 489-496
24. Shi F., Gounko N.V., Wang X., Ronken E., and Hoekstra D. In situ entry of oligonucleotides into brain cells can occur through a nucleic acid channel. Oligonucleotides 17 (2007) 122-133
25. Lorenz P., Misteli T., Baker B.F., Bennett C.F., and Spector D.L. Nucleocytoplasmic shuttling: a novel in vivo property of antisense phosphorothioate oligodeoxynucleotides. Nucleic Acids Res 28 (2000) 582-592
26. Rifai A., Byrsch W., Fadden K., Clark J., and Schlingensipen K.-H. Clearance kinetics, biodistribution, and organ saturability of phosphorothioate oligodeoxynucleotides in mice. Am J Pathol 149 (1996) 717-725
27. Phillips J.A., Craig S.J., Bayley D., Christian R.A., Geary R.S., and Nicklin P.L. Pharmacokinetics, metabolism and elimination of a 20-mer phosphorothioate oligodeoxynucleotide (CGP 69846A) after intravenous and subcutaneous administration. Biochem Pharmacol (Elsevier) 54 (1997) 657-668
28. Geary R.S., Leeds J.M., Fitchett J., Burckin T., Troung L., Spainhour C., et al. Pharmacokinetics and metabolism in mice of a phosphorothioate oligonucleotide antisense inhibitor of C-raf-1 kinase expression. Drug Metab Dispos 25 (1997) 1272-1281
29. Graham M.J., Crooke S.T., Monteith D.K., Cooper S.R., Lemonidis K.M., Stecker K.K., et al. In vivo distribution and metabolism of a phosphorothioate oligonucleotide within rat liver after intravenous administration. J Pharmacol Exp Ther 286 (1998) 447-458
30. Graham M.J., Crooke S.T., Lemonidis K.M., Gaus H.J., Templin M.V., and Crooke R.M. Hepatic distribution of a phosphorothioate oligodeoxynucleotide within rodents following intravenous administration. Biochem Pharmacol 62 (2001) 297-306
31. Yu R.Z., Zhang H., Geary R.S., Graham M., Masarjian L., Lemonidis K., et al. Pharmacokinetics and pharmacodynamics of an antisense phosphorothioate oligonucleotide targeting Fas mRNA in mice. J Pharmacol Exp Ther 296 (2001) 388-395
32. Butler M., McKay R.A., Popoff I.J., Gaarde W.A., Witchell D., Murray S.F., et al. Specific inhibition of PTEN expression reverses hyperglycemia in diabetic mice. Diabetes 51 (2002) 1028-1034
33. Baker B.F., Lot S.S., Condon T.P., Cheng-Flournoy S., Lesnik E.A., Sasmor H.M., et al. 2′-O-(2-methoxy)ethyl-modified anti-intercellular adhesion molecule 1 (ICAM-1) oligonucleotides selectively increase the ICAM-1 mRNA level and inhibit formation of the ICAM-1 translation initiation complex in human umbilical vein endothelial cells. J Biol Chem 272 (1997) 11994-12000
34. Leeds J.M., Graham M.J., Troung L., and Cummins L.L. Quantitation of phosphorothioate oligonucleotides in human plasma. Anal Biochem 235 (1996) 36-43
35. Geary R.S., Matson J., and Levin A.A. A nonradioisotope biomedical assay for intact oligonucleotide and its chain-shortened metabolites used for determination of exposure and elimination half-life of antisense drugs in tissue. Anal Biochem 274 (1999) 241-248
36. Yu R.Z., Geary R.S., and Levin A.A. Application of novel quantitative bioanalytical methods for pharmacokinetic and pharmacokinetic/pharmacodynamic assessments of antisense oligonucleotides. Curr Opin Drug Discov Devel 7 (2004) 195-203
37. Zhang H., Cook J., Nickel J., Yu R., Stecker K., Myers K., et al. Reduction of liver Fas expression by an antisense oligonucleotide protects mice from fulminant hepatitis. Nat Biotechnol 18 (2000) 862-867
38. Yu R.Z., Geary R.S., and Levin A.A. Pharmacokinetics and pharmacodynamics of antisense oligonucleotides. In: Meyers R.A. (Ed). Encyclopedia of molecular cell biology and molecular medicine (2007), Wiley-VCH, Weinheim, Germany
39. Brown D.A., Kang S.H., Gryaznov S.M., DeDionisio L., Heidenreich O., Sullivan S., et al. Effect of phosphorothioate modification of oligodeoxynucleotides on specific protein binding. J Biol Chem 269 (1994) 26801-26805
40. Watanabe T.A., Geary R.S., and Levin A.A. Plasma protein binding of an antisense oligonucleotide targeting human ICAM-1 (ISIS 2302). Oligonucleotides 16 (2006) 169-180
41. Butler M., Stecker K., and Bennett C.F. Cellular distribution of phosphorothioate oligodeoxynucleotides in normal rodent tissues. Lab Invest 77 (1997) 379-388
42. Butler M., Stecker K., and Bennett C.F. Histological localization of phosphorothioate oligodeoxynucleotides in normal rodent tissue. Nucleosides Nucleotides 16 (1997) 1761-1764
43. Molitoris B.A., and Sandoval R.M. Pharmacophotonics: utilizing multi-photon microscopy to quantify drug delivery and intracellular trafficking in the kidney. Adv Drug Delivery Rev 58 (2006) 809-823
44. Mager D.E. Target-mediated drug disposition and dynamics. Biochem Pharmacol 72 (2006) 1-10
45. Mager D.E., and Jusko W.J. General pharmacokinetic model for drugs exhibiting target-mediated drug disposition. J Pharmacokinet Pharmacodyn 28 (2001) 507-532
46. Hanss B., Leal-Pinto E., Bruggeman L.A., Copeland T.D., and Klotman P.E. Identification and characterization of a cell membrane nucleic acid channel. Proc Natl Acad Sci USA 95 (1998) 1921-1926
47. Hanss B., Leal-Pinto E., Teixeira A., Christian R.E., Shabanowitz J., Hunt D.F., et al. Cytosolic malate dehydrogenase confers selectivity of the nucleic acid-conducting channel. Proc Natl Acad Sci USA 99 (2002) 1707-1712
48. Yu R.Z., Baker B., Chappell A., Geary R.S., Chueng E., and Levin A.A. Development of an ultrasensitive noncompetitive hyridization-ligation enzyme-linked immunosorbent assay for the determination of phosphorothioate oligodeoxynucleotide in plasma. Anal Biochem 304 (2002) 19-25